Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency von Punwani, Divya, Kawahara, Misako, Yu, Jason, Sanford, Ukina, Roy, Sushmita, Patel, Kiran, Carbonaro, Denise A, Karlen, Andrea D, Khan, Sara, Cornetta, Kenneth, Rothe, Michael, Schambach, Axel, Kohn, Donald B, Malech, Harry L, McIvor, R Scott, Puck, Jennifer M, Cowan, Morton J

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Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells von Smith, Miles C, Belur, Lalitha R, Karlen, Andrea D, Erlanson, Olivia, Podetz-Pedersen, Kelly M, McKenzie, Jessica, Detellis, Jenn, Gagnidze, Khatuna, Parsons, Geoffrey, Robinson, Nicholas, Labarre, Shelby, Shah, Saumil, Furcich, Justin, Lund, Troy C, Tsai, Hsing-Chen, McIvor, R Scott, Bonner, Melissa

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Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II von Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Podetz-Pedersen, Kelly, Erlanson, Olivia, Laoharawee, Kanut, Furcich, Justin, Lund, Troy C., You, Yun, Seelig, Davis, Webber, Beau R., McIvor, R. Scott

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Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I von Belur, Lalitha R., Huber, Avery K., Mantone, Hillary, Robertson, Mason, Smith, Miles C., Karlen, Andrea D., Kitto, Kelley F., Ou, Li, Whitley, Chester B., Braunlin, Elizabeth, Furcich, Justin, Lund, Troy C., Seelig, Davis, Fairbanks, Carolyn A., Buss, Nicholas, Kim, Kwi Hye, McIvor, R. Scott

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Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice von Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Erlanson, Olivia, Furcich, Justin, Lund, Troy C., Seelig, Davis, Kitto, Kelley F., Fairbanks, Carolyn A., Kim, Kwi Hye, Buss, Nick, McIvor, R. Scott

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AAV-mediated gene transfer to colon-innervating primary afferent neurons von Gore, Reshma, Esmail, Tina, Pflepsen, Kelsey, Marron Fernandez de Velasco, Ezequiel, Kitto, Kelley F, Riedl, Maureen S, Karlen, Andrea, McIvor, R Scott, Honda, Christopher N, Fairbanks, Carolyn A, Vulchanova, Lucy

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Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency von Multhaup, Megan M, Podetz-Pedersen, Kelly M, Karlen, Andrea D, Olson, Erik R, Gunther, Roland, Somia, Nikunj V, Blazar, Bruce R, Cowan, Morton J, McIvor, R Scott

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Cytotoxicity Associated with Artemis Overexpression After Lentiviral Vector-Mediated Gene Transfer von MULTHAUP, Megan, KARLEN, Andrea D, SWANSON, Debra L, WILBER, Andrew, SOMIA, Nikunj V, COWAN, Morton J, MCLVOR, R. Scott

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Identification of inflammatory cells in dilated ascending aortas of IDUA-deficient (MPS I) mice von Binstadt, Bryce, Seelig, Davis, Auger, Jennifer, Karlen, Andrea, Evans, Michael, McIvor, R. Scott, Braunlin, Elizabeth

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Characterization of the human artemis promoter by heterologous gene expression in vitro and in vivo von Multhaup, Megan M, Gurram, Sweta, Podetz-Pedersen, Kelly M, Karlen, Andrea D, Swanson, Debra L, Somia, Nikunj V, Hackett, Perry B, Cowan, Morton J, McIvor, R Scott

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Comparative systemic and neurologic effectiveness of intravenous and intrathecal AAV9 delivered individually or combined in a murine model of mucopolysaccharidosis type I von Belur, Lalitha, Huber, Avery, Mantone, Hillary, Karlen, Andrea, Smith, Miles, Ou, Li, Whitley, Chester B., Buss, Nicholas, McIvor, R. Scott

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Comparative effectiveness of intravenous and intrathecal AAV9.CB7.hIDS (RGX-121) in a murine model of mucopolysaccharidosis type II von Smith, Miles C., Belur, Lalitha, Erlanson, Olivia L., Furcich, Justin W., Karlen, Andrea D., Lund, Troy C., Buss, Nicholas, McIvor, R. Scott

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Treatment of cardiac, neurologic, and skeletal manifestations of murine MPS I with AAV9-IDUA: Efficacy study of vector dose and route of administration von Belur, Lalitha, Huber, Avery, Mantone, Hillary, Robertson, Mason, Smith, Miles, Karlen, Andrea, Braunlin, Elizabeth, Lund, Troy, Seelig, Davis, Buss, Nicholas, McIvor, R. Scott

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Effect of supraphysiological alpha-L-iduronidase (IDUA) expression on skeletal manifestations in mucopolysaccharidosis type I (MPS I) mice following ex vivo lentiviral vector trans... von Lund, Troy C., Belur, Lalitha, Huber, Avery, Mantone, Hillary, Smith, Miles, Podetz-Pedersen, Kelly, Karlen, Andrea, Braunlin, Elizabeth, Robinson, Nick, Tsai, Hsing-Chen, McIvor, R. Scott, Bonner, Melissa

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Ex vivo lentiviral transduction of hematopoietic stem cells in mucopolysaccharidosis type II (MPS II) mice achieves high levels of systemic iduronate-2-sulfatase (IDS) enzyme activ... von Smith, Miles C., Belur, Lalitha, Erlanson, Olivia, Podetz-Pedersen, Kelly, Karlen, Andrea, McKenzie, Jessica, Detellis, Jenn, Gagnidze, Khatuna, Parsons, Geoff, Robinson, Nick, Labarre, Shelby, Scarglia, Gina, Shah, Saumil, Tsai, Hsing-Chen, McIvor, R. Scott, Bonner, Melissa

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Systemic high-level IDUA enzyme activity with correction of neurologic deficit in mucopolysaccharidosis type I mice by lentiviral transduction of hematopoietic stem cells von Belur, Lalitha, McKenzie, Jessica, Podetz-Pedersen, Kelly, Karlen, Andrea, de Souza, Patricia Coutinho, LaBarre, Shelby, Smith, Miles, Huber, Avery, Detellis, Jennifer, Gagnidze, Khatuna, Parsons, Geoff, Tsai, Hsing-Chen, McIvor, R. Scott, Bonner, Melissa

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Systemic high-level IDUA enzyme activity with correction of neurologic deficit in mucopolysaccharidosis type I mice by ex vivolentiviral transduction of hematopoietic stem cells von Belur, Lalitha, McKenzie, Jessica, Podetz-Pedersen, Kelly, Karlen, Andrea, de Souza, Patricia Coutinho, LaBarre, Shelby, Smith, Miles, Huber, Avery, Detellis, Jennifer, Gagnidze, Khatuna, Parsons, Geoff, Tsai, Hsing-Chen, McIvor, R. Scott, Bonner, Melissa

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Prevention of Cardiac, Neurologic, and Skeletal Manifestations of Murine MPS I by Either Systemic or CSF-Directed AAV9-IDUA/RGX-111 Administered at Minimal Effective Dose von Belur, Lalitha R., Huber, Avery K., Mantone, Hillary, Robertson, Mason, Smith, Miles C., Karlen, Andrea D., Kitto, Kelley F., Ou, Li, Whitley, Chester B., Braunlin, Elizabeth, Furcich, Justin, Lund, Troy C., Seelig, Davis, Fairbanks, Carolyn A., Buss, Nicholas, Kim, Kwi Hye, McIvor, R. Scott

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Gene therapy for Hunter syndrome: Prevention of neurocognitive deficit in MPS II mice by engraftment with lentiviral vector-transduced hematopoietic stem cells (HSC) but not with w... von McIvor, R. Scott, Podetz-Pedersen, Kelly, Nan, Zhenhong, Karlen, Andrea, Slepushkin, Vladimir, Roy, Andre, Yakovlev, Galina, Gong, Zi, Muenzer, Joseph, Dropulic, Boro

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Hodgkin lymphoma: hypodense lesions in mediastinal masses von Damek, Adrian, Kurch, Lars, Franke, Friedrich Christian, Attarbaschi, Andishe, Beishuizen, Auke, Cepelova, Michaela, Ceppi, Francesco, Daw, Stephen, Dieckmann, Karin, Fernández-Teijeiro, Ana, Feuchtinger, Tobias, Flerlage, Jamie E., Fosså, Alexander, Georgi, Thomas W., Hasenclever, Dirk, Hraskova, Andrea, Karlen, Jonas, Klekawka, Tomasz, Kluge, Regine, Körholz, Dieter, Landman-Parker, Judith, Leblanc, Thierry, Mauz-Körholz, Christine, Metzler, Markus, Pears, Jane, Steglich, Jonas, Uyttebroeck, Anne, Vordermark, Dirk, Wallace, William Hamish, Wohlgemuth, Walter Alexander, Stoevesandt, Dietrich

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